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List of works by Mario Amendola

A Double-Switch Vector System Positively Regulates Transgene Expression by Endogenous microRNA Expression (miR-ON Vector) ⬇️

scientific article published on February 26, 2013

A combination of cyclophosphamide and interleukin-2 allows CD4+ T cells converted to Tregs to control scurfy syndrome

scientific article published on 01 April 2021

Author Correction: Ex vivo editing of human hematopoietic stem cells for erythroid expression of therapeutic proteins

scientific article published on 13 August 2020

Base-editing-mediated dissection of a γ-globin cis-regulatory element for the therapeutic reactivation of fetal hemoglobin expression

scientific article published on 4 November 2022

Coordinate dual-gene transgenesis by lentiviral vectors carrying synthetic bidirectional promoters.

scientific article

Correction of β-thalassemia by CRISPR/Cas9 editing of the α-globin locus in human hematopoietic stem cells

scientific article published on 01 March 2021

Easy quantitative assessment of genome editing by sequence trace decomposition

scientific article

Editing a γ-globin repressor binding site restores fetal hemoglobin synthesis and corrects the sickle cell disease phenotype

scientific article published on 12 February 2020

Effects of phosphorylation and neuronal activity on the control of synapse formation by synapsin I

scientific article published on 01 November 2011

Efficient Tet-dependent expression of human factor IX in vivo by a new self-regulating lentiviral vector

scientific article

Endogenous microRNA can be broadly exploited to regulate transgene expression according to tissue, lineage and differentiation state

scientific article published on 16 November 2007

Ex vivo editing of human hematopoietic stem cells for erythroid expression of therapeutic proteins

scientific article published on 29 July 2020

Forebrain ependymal cells are Notch-dependent and generate neuroblasts and astrocytes after stroke

scientific article

Forkhead box protein 3 (FOXP3) mutations lead to increased TH17 cell numbers and regulatory T-cell instability

scientific article published on 13 October 2011

Generation of potent and stable human CD4+ T regulatory cells by activation-independent expression of FOXP3.

scientific article published on 6 November 2007

Genome-wide maps of nuclear lamina interactions in single human cells

scientific article

Identification of hematopoietic stem cell-specific miRNAs enables gene therapy of globoid cell leukodystrophy

scientific article published in November 2010

Induction of fetal hemoglobin synthesis by CRISPR/Cas9-mediated editing of the human β-globin locus.

scientific article published on 8 March 2018

Mechanisms and dynamics of nuclear lamina-genome interactions

scientific article published on 30 March 2014

Nuclear lamins are not required for lamina-associated domain organization in mouse embryonic stem cells

scientific article published on 17 March 2015

Optimization of CRISPR/Cas9 Delivery to Human Hematopoietic Stem and Progenitor Cells for Therapeutic Genomic Rearrangements

scientific article published on 17 October 2018

Oxidative stress and cell death in cells expressing L-ferritin variants causing neuroferritinopathy

scientific article published on 23 September 2009

Regulated and multiple miRNA and siRNA delivery into primary cells by a lentiviral platform

scientific article published on 17 March 2009

Reprogramming T lymphocytes for melanoma adoptive immunotherapy by T-cell receptor gene transfer with lentiviral vectors

scientific article published in December 2009

Single-cell dynamics of genome-nuclear lamina interactions

scientific article published on 21 March 2013

Stable knockdown of microRNA in vivo by lentiviral vectors

scientific article

The Cohesin Release Factor WAPL Restricts Chromatin Loop Extension

scientific article

Widespread enzymatic correction of CNS tissues by a single intracerebral injection of therapeutic lentiviral vector in leukodystrophy mouse models

scientific article published on 4 March 2010

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